Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy

Autosomal Dominant Polycystic Kidney Disease Clinical Trial

Official title:

Rapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00920309
• Other study ID #: HIC#0903004934
• Status: Terminated
• Phase: Phase 2/Phase 3
• First received: June 12, 2009
• Last updated: March 7, 2014
• Start date: June 2009
• Est. completion date: July 2010

Study information

• Verified date: March 2014
• Source: Yale University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

Currently the only approved use for rapamycin (sirolimus) is for immunosuppression after renal transplantation.

This trial is designed to determine whether rapamycin is safe and effective treatment for patients with polycystic kidney disease (ADPKD). Patients will be followed by volumetric magnetic resonance imaging (MRI) to observe for change in kidney (and cyst) size. Blood and urine samples will also be collected to evaluate for change in biomarkers with treatment.

Description:

This is a 2 year, open label trial to evaluate the role of rapamycin as treatment for ADPKD. Patients will be randomized 2:1 to the rapamycin arm or to standard therapy. The dose of rapamycin will be adjusted so that patients obtain 24 trough levels of 4-6ng/ml. There will be a volumetric MRI measurement at the start and end of the treatment period. Patients will be monitored every 4 months throughout the study.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 21
• Est. completion date: July 2010
• Est. primary completion date: July 2010
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 18 Years to 70 Years

Eligibility

Inclusion Criteria:

• adult ADPKD patients aged 18-70

• combined kidney volume >1200 ml

• estimated creatinine clearance >60 ml/min

• absence of implanted ferromagnetic objects

Exclusion Criteria:

• Age >70

• uncontrolled hyperlipidemia

• Proteinuria >500 mg/day

• unstable cerebral aneurysm

• active coronary artery disease

• diagnosis of another systemic condition affecting kidney function (ie: diabetes, hepatitis B or C, HIV)

• diagnosis of cancer other than skin cancer

• pregnancy or lactation

• presence of implanted ferromagnetic objects

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Autosomal Dominant Polycystic Kidney Disease
• Kidney Diseases
• Polycystic Kidney Diseases
• Polycystic Kidney, Autosomal Dominant

Intervention

Drug:
• Rapamycin
The starting dose of rapamycin will be 1 mg daily. The dose will be increased as needed to achieve a 24 hour trough level of 4-6 ng/ml.

Other:
• Standard of Care-Placebo

Locations

Country	Name	City	State
United States	Yale Center for Clinical Investigation	New Haven	Connecticut

Sponsors (1)

Lead Sponsor	Collaborator
Yale University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Total Kidney Volume (mL)		2 years	No
Secondary	Glomerular Filtration Rate (Kidney Function)		2 years	No